Future Directions in the Treatment of Neuroendocrine Tumors: Consensus Report of the National Cancer Institute Neuroendocrine Tumor Clinical Trials Planning Meeting
Future Directions in the Treatment of Neuroendocrine Tumors: Consensus Report of the National Cancer Institute Neuroendocrine Tumor Clinical Trials Planning Meeting
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DOI：
10.1200/jco.2010.33.2056
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发表时间：
2011-03-01
影响因子：
45.3
通讯作者：
Yao, James C.
中科院分区：
文献类型：
--
作者：
Kulke, Matthew H.;Siu, Lillian L.;Yao, James C.
神经内分泌肿瘤（NETs）产生于各种解剖部位，并具有产生激素和血管活性肽的能力。由于它们被认为是罕见的，NET在历史上并没有成为严格的临床研究的焦点。然而，NET的诊断发病率一直在增加，估计在美国的患病率超过10万人。最近完成的几项III期研究，包括评估奥曲肽，舒尼替尼和依维莫司的研究，已经证明严格评估这种疾病的新药是可行的，并且可以导致实践改变的结果。美国国家癌症研究所GI指导委员会的NET工作组召开了一次临床试验计划会议，以确定关键的未满足的需求，制定适当的研究终点，标准化临床试验纳入标准，并为美国合作组计划制定未来NET研究的优先事项。重点放在制定设计良好的临床试验，明确定义的疗效标准。主要建议包括将胰腺NET与其他部位的NET分开评价，并将组织学分化差的患者排除在低级别组织学试验之外。评价控制激素综合征的新药的研究应尽可能避免生长抑素类似物洗脱期，并应包括生活质量终点。由于观察到许多患者进展后的长期生存期，因此建议将无进展生存期作为III期研究和II期研究的可行且相关的主要终点，其中在无放射学缓解的情况下，预计进展延迟。J Clin Oncol 29：934-943. (C)2011年美国临床肿瘤学会
Neuroendocrine tumors (NETs) arise from a variety of anatomic sites and share the capacity for production of hormones and vasoactive peptides. Because of their perceived rarity, NETs have not historically been a focus of rigorous clinical research. However, the diagnosed incidence of NETs has been increasing, and the estimated prevalence in the United States exceeds 100,000 individuals. The recent completion of several phase III studies, including those evaluating octreotide, sunitinib, and everolimus, has demonstrated that rigorous evaluation of novel agents in this disease is both feasible and can lead to practice-changing outcomes. The NET Task Force of the National Cancer Institute GI Steering Committee convened a clinical trials planning meeting to identify key unmet needs, develop appropriate study end points, standardize clinical trial inclusion criteria, and formulate priorities for future NET studies for the US cooperative group program. Emphasis was placed on the development of well-designed clinical trials with clearly defined efficacy criteria. Key recommendations include the evaluation of pancreatic NET separately from NETs of other sites and the exclusion of patients with poorly differentiated histologies from trials focused on low-grade histologies. Studies evaluating novel agents for the control of hormonal syndromes should avoid somatostatin analog washout periods when possible and should include quality-of-life end points. Because of the observed long survival after progression of many patients, progression-free survival is recommended as a feasible and relevant primary end point for both phase III studies and phase II studies where a delay in progression is expected in the absence of radiologic responses. J Clin Oncol 29: 934-943. (C) 2011 by American Society of Clinical Oncology